ARDSLEY, N.Y.--(BUSINESS WIRE)--
Acorda Therapeutics, Inc. (Nasdaq:ACOR)
today announced Phase 3 clinical data of CVT-301, showing a
statistically significant improvement in motor function in people with
Parkinson’s disease experiencing OFF periods. CVT-301 is an
investigational, inhalable formulation of levodopa (L-dopa). It is being
studied as a treatment for OFF periods in people with Parkinson’s
disease taking an oral carbidopa / levodopa regimen. OFF periods refer
to the re-emergence of Parkinson’s symptoms.

“We are greatly encouraged by the efficacy and safety results of this
trial, which validate the positive Phase 2b results,” said Burkhard
Blank, M.D., Chief Medical Officer of Acorda. “We would like to express
our gratitude to the study volunteers and clinical investigators who
participated in this trial to advance our understanding of this
potentially important therapy for people with Parkinson’s.”

The safety profile of CVT-301 in this study was consistent with that
observed in the Phase 2b trial. Spirometry and diffusing capacity of the
lung for carbon monoxide (DLCO) tests showed no notable
pulmonary safety signals. The Company is currently conducting two
studies to assess the long-term safety profile of CVT-301. Up to
12-month data from these studies are expected by the end of the first
quarter of 2017.

The Company plans to file a New Drug Application (NDA) in the United
States by the end of the second quarter of 2017, pending results of the
long-term safety studies. The Company also plans to file a Marketing
Authorization Application (MAA) in Europe by the end of 2017, pending
additional data analyses.

Peter LeWitt, M.D., M.Med.Sc., Director of the PD and Movement Disorders
Program at Henry Ford Hospital and lead investigator of the study said,
“The re-emergence of Parkinson’s disease symptoms has a major negative
impact on the lives of people with this disease, as well as on their
families and care partners. Managing symptoms of OFF periods continues
to be a significant unmet need for people taking oral carbidopa/levodopa
regimens. Delivering levodopa by the pulmonary route offers an important
treatment option for people with Parkinson’s disease.”

Detailed trial results will be presented at a future medical meeting.

SPAN-PD Safety Findings

Participants reporting serious adverse events (SAEs) were as follows: 3
(2.7%) in the placebo arm, 6 (5.3%) in the 60 mg arm, and 2 (1.8%) in
the 84 mg arm. There was one death in the study, a suicide in the 60 mg
group, judged by the investigator to be not related to study drug.

The most common adverse events that were reported in any study arm at
>5% were:

Adverse Event

n (%)

Placebo

(N=112)

CVT-301 60 mg

(N=113)

CVT-301 84 mg (N=114)

Cough

2 (1.8%)

17 (15.0%)

17 (14.9%)

Upper Respiratory Tract Infection

3 (2.7%)

2 (1.8%)

7 (6.1%)

Throat Irritation

0

8 (7.1%)

1 (0.9%)

Nausea

3 (2.7%)

0

6 (5.3%)

Sputum Discolored

0

0

6 (5.3%)

When cough was reported, it was typically mild and reported once per
participant during the course of treatment. Three of 227 participants
receiving CVT-301 discontinued the study due to cough.

SPAN-PD Trial Design

The Phase 3, randomized, double-blind, placebo-controlled clinical trial
evaluated the efficacy and safety of CVT-301 compared with placebo in
people with Parkinson’s who experience motor fluctuations (OFF periods).
All participants were on a stable regimen of oral carbidopa / levodopa,
and were also maintained on their other existing Parkinson’s therapies.
A total of 339 study participants were randomized and received at least
one dose of CVT-301 or placebo. Participants self-administered treatment
up to five times daily for 12 weeks.

The primary endpoint of the study was the change at Week 12 in Unified
Parkinson’s Disease Rating Scale-Part 3 (UPDRS III) score relative to
placebo at 30 minutes post-treatment for the 84 mg dose. Key secondary
endpoints, measured at Week 12, included: proportion of participants
achieving an ON state within 60 minutes of treatment and maintained at
60 minutes; change in UPDRS III score at 10 and 20 minutes following
treatment; Patient Global Impression of Change (PGI-C) improvement; and
total daily OFF time as recorded in participant diary.

About Parkinson’s Disease and OFF Periods

Approximately one million people in the U.S. and 1.2 million Europeans
are diagnosed with Parkinson’s disease (PD); OFF periods are experienced
by approximately 350,000 in the U.S. and 420,000 in Europe.

Parkinson’s is a progressive neurodegenerative disorder resulting from
the gradual loss of certain neurons responsible for producing dopamine.
It causes a range of symptoms including impaired movement, muscle
stiffness and tremors. As PD progresses, people with Parkinson’s
experience OFF periods, which are characterized by the re-emergence of
PD symptoms. This re-emergence can occur even when an individual’s
treatment regimen has been optimized.

OFF periods can be very disruptive to the lives of people with
Parkinson’s, their families and caregivers. OFF periods can increase in
frequency and severity during the course of the disease.

About CVT-301 and ARCUS®

CVT-301 is being developed as a self-administered, inhaled levodopa
(L-dopa) therapy for the treatment of symptoms of OFF periods in people
with Parkinson’s disease taking an oral carbidopa / levodopa regimen.

CVT-301 utilizes Acorda’s investigational ARCUS® platform for
inhaled therapeutics. CVT-301 delivers a precise dose of a dry powder
formulation of L-dopa to the lung. Oral medication can be associated
with slow and variable onset of action, as the medicine is absorbed
through the gastrointestinal (digestive) tract before reaching the
brain. Inhaled treatments enter the body through the lungs and reach the
brain shortly thereafter, bypassing the digestive system.

About Acorda Therapeutics

Founded in 1995, Acorda Therapeutics is a biotechnology company focused
on developing therapies that restore function and improve the lives of
people with neurological disorders.

For more information, please visit the Company’s website at: www.acorda.com.

Forward-Looking Statement

This press release includes forward-looking statements. All statements,
other than statements of historical facts, regarding management's
expectations, beliefs, goals, plans or prospects should be considered
forward-looking. These statements are subject to risks and uncertainties
that could cause actual results to differ materially, including: the
ability to complete the Biotie transaction on a timely basis; the
ability to realize the benefits anticipated from the Biotie and Civitas
transactions, among other reasons because acquired development programs
are generally subject to all the risks inherent in the drug development
process and our knowledge of the risks specifically relevant to acquired
programs generally improves over time; the ability to successfully
integrate Biotie’s operations and Civitas’ operations, respectively,
into our operations; we may need to raise additional funds to finance
our expanded operations and may not be able to do so on acceptable
terms; our ability to successfully market and sell Ampyra
(dalfampridine) Extended Release Tablets, 10 mg in the U.S.; third party
payers (including governmental agencies) may not reimburse for the use
of Ampyra or our other products at acceptable rates or at all and may
impose restrictive prior authorization requirements that limit or block
prescriptions; the risk of unfavorable results from future studies of
Ampyra or from our other research and development programs, including
CVT-301 or any other acquired or in-licensed programs; we may not be
able to complete development of, obtain regulatory approval for, or
successfully market CVT-301, any other products under development, or
the products that we will acquire when we complete the Biotie
transaction; the occurrence of adverse safety events with our products;
delays in obtaining or failure to obtain and maintain regulatory
approval of or to successfully market Fampyra outside of the U.S. and
our dependence on our collaborator Biogen in connection therewith;
competition; failure to protect our intellectual property, to defend
against the intellectual property claims of others or to obtain third
party intellectual property licenses needed for the commercialization of
our products; and failure to comply with regulatory requirements could
result in adverse action by regulatory agencies.

These and other risks are described in greater detail in our filings
with the Securities and Exchange Commission. We may not actually achieve
the goals or plans described in our forward-looking statements, and
investors should not place undue reliance on these statements.
Forward-looking statements made in this press release are made only as
of the date hereof, and we disclaim any intent or obligation to update
any forward-looking statements as a result of developments occurring
after the date of this press release.